Comparison of post-authorisation measures from regulatory authorities with additional evidence requirements from the HTA body in Germany - are additional data requirements by the Federal Joint Committee justified?

Jörg Ruof,Charalabos-Markos Dintsios,Thomas Staab,Friedrich Wilhelm Schwartz,Jakob Schröter

doi:10.1186/s13561-016-0124-4

Abstract

ObjectivesThe aim of this study was to compare post-authorisation measures (PAMs) from the European Medicines Agency (EMA) with data requests in fixed-termed conditional appraisals of early benefit assessments from the German Federal Joint Committee (G-BA).MethodsMedicinal products with completed benefit assessments during an assessment period of 3.5 years were considered. PAMs extracted from European Public Assessment Reports (EPARs) were compared with data requests issued by the G-BA in the context of conditional appraisals.ResultsTwenty conditional appraisals (19 products) and 34 EPARs containing PAMs (33 products) were identified. Data categories (efficacy, safety, etc.), data types (type of study required to address the request) and clarity of requests were determined. Conditional appraisals disproportionately focused on oncology products (13/19 products with conditional appraisals vs. 14/33 products with PAMs). No clear rationale for the G-BA issuing conditional appraisals could be identified in public sources. Both EMA and G-BA requested mainly efficacy and safety data (44/54 and 23/35 categories requested, respectively); however, 28/35 G-BA data requirements went beyond requests made by the EMA. Almost half of the G-BA requests (9/20), but no PAMs, were unclear, and no methodological guidance for fulfilling the data requirements was provided by the G-BA.ConclusionsBetter alignment between data requests from regulatory authorities and health technology assessment bodies is strongly recommended.

Highlights

At the time of marketing authorisation, the available information relating to a medicine may not yet be sufficient to fully assess the benefit/risk profile to the desired degree of certainty or may lack aspects of interests to the Committee for Medicinal Products for Human Use (CHMP) that are requested to be provided after marketing authorisation is granted
Analysis set The analysis included 77 early benefit assessments conducted during the 3.5-years assessment period and their corresponding European Public Assessment Report (EPAR)/EPAR variations where available
67 products with 74 EPARs/EPAR variations were included in the Post-authorisation measure (PAM) analysis

Summary

Introduction

At the time of marketing authorisation, the available information relating to a medicine may not yet be sufficient to fully assess the benefit/risk profile to the desired degree of certainty or may lack aspects of interests to the Committee for Medicinal Products for Human Use (CHMP) that are requested to be provided after marketing authorisation is granted. The FDA refers to these additional requirements as post-marketing requirements and commitments These gather additional information about a product's safety, efficacy or optimal use [1]. The EMA can require postauthorisation measures (PAMs) if the Agency’s committees consider the generation of additional data necessary from a “public health perspective to complement the available data with additional data about the safety and, in certain cases, the efficacy of authorised medicinal products”. Like their FDA equivalent, PAMs are mainly required in order to answer open questions regarding the risk/benefit profile or safety concerns of a product [2]

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